FV 953. Parents’ Experiences during the Compassionate Use Program (Nusinersen) for Patients with Spinal Muscular Atrophy Type 1—A Qualitative Interview Study

2018 ◽  
Author(s):  
Thorsten Langer ◽  
Petra Kiefer ◽  
Astrid Pechmann ◽  
Janbernd Kirschner
Keyword(s):  
Spinal Muscular Atrophy ◽  
Muscular Atrophy ◽  
Qualitative Interview ◽  
Interview Study ◽  
Compassionate Use ◽  
Spinal Muscular Atrophy Type ◽  
Qualitative Interview Study ◽  
Parents Experiences

Long‐term benefits of nusinersen in a child affected by cystic fibrosis and spinal muscular atrophy type 1

2021 ◽  
Author(s):  
Giorgia Bruno ◽  
Bernadette Donnarumma ◽  
Alessia Inverardi ◽  
Paolo Buonpensiero ◽  
Angela Sepe ◽  
...  
Keyword(s):  
Cystic Fibrosis ◽  
Spinal Muscular Atrophy ◽  
Muscular Atrophy ◽  
Spinal Muscular Atrophy Type

scholarly journals Nusinersen in spinal muscular atrophy type 1 from neonates to young adult: 1-year data from three Asia-Pacific regions

2021 ◽  
pp. jnnp-2020-324532
Author(s):  
Sophelia Hoi-Shan Chan ◽  
Jong-Hee Chae ◽  
Yin-Hsiu Chien ◽  
Tae-Sung Ko ◽  
Jee Hun Lee ◽  
...  
Keyword(s):  
Young Adult ◽  
Spinal Muscular Atrophy ◽  
Muscular Atrophy ◽  
Asia Pacific ◽  
Spinal Muscular Atrophy Type

scholarly journals Choosing Life with Spinal Muscular Atrophy Type 1

2020 ◽  
Vol 37 (5) ◽  
pp. 1708-1713
Author(s):  
Moran Lavie ◽  
Hodaya Nisnkorn ◽  
Liora Sagi ◽  
Israel Amirav
Keyword(s):  
Spinal Muscular Atrophy ◽  
Muscular Atrophy ◽  
Spinal Muscular Atrophy Type

First experience of Nusinersen early access program in patients with spinal muscular atrophy type 1

2017 ◽  
Vol 27 ◽  
pp. S211-S212 ◽  
Author(s):  
E. Gargaun ◽  
K. Aragon-Gawinska ◽  
A. Seferian ◽  
T. Gidaro ◽  
S. Gilabert ◽  
...  
Keyword(s):  
Spinal Muscular Atrophy ◽  
Muscular Atrophy ◽  
Spinal Muscular Atrophy Type ◽  
Early Access ◽  
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